Invention Application
- Patent Title: RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES
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Application No.: US18786394Application Date: 2024-07-26
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Publication No.: US20240392303A1Publication Date: 2024-11-28
- Inventor: Prashant Mali , Dhruva Katrekar
- Applicant: The Regents of the University of California
- Applicant Address: US CA Oakland
- Assignee: The Regents of the University of California
- Current Assignee: The Regents of the University of California
- Current Assignee Address: US CA Oakland
- Main IPC: C12N15/115
- IPC: C12N15/115 ; A61K38/00 ; A61P21/00 ; C12N9/78 ; C12N15/113

Abstract:
Aspects of the disclosure relate to a gene therapy approach for diseases, disorders, or conditions caused by mutation in the stop codon utilizing modified tRNA. At least 10-15% of all genetic diseases, including muscular dystrophy (e.g. Duchene muscular dystrophy), some cancers, beta thalassemia, Hurler syndrome, and cystic fibrosis, fall into this category. Not to be bound by theory, it is believed that this approach is safer than CRISPR approaches due to minimal off-target effects and the lack of genome level changes.
Information query
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